Evaluation of PKU Sphere

NCT03419819 | Completed

• 1 other identifier: SCT-PKUS-2016-11-28
• Study Type: interventional
• Target: 28
• Locations: 1 country
• Sites: 1

Brief Summary

A prospective, open-label study of PKU Sphere in patients with PKU following a phenylalanine restricted therapeutic diet.

Conditions

Phenylketonurias

Keywords

Phenylketonuria; GMP; Sphere; Child; Adult; Phenylalanine; PKU; Glycomacropeptide

Outcome Measures

Primary Outcomes (15)

• Phase 1: taste

  Questionnaire data captured to evaluate taste

  Days 1 - 7.

• Phase 1: smell

  Questionnaire data captured to evaluate smell

  Days 1 - 7.

• Phase 1: texture

  Questionnaire data captured to evaluate texture

  Days 1 - 7.

• Phase 1: gastrointestinal tolerance

  Questionnaires will be completed daily during the 1-week taste test.

  Days 1 - 7.

• Phase 1: phenylalanine concentration

  Phenylalanine concentrations measured via blood spot analysis.

  Days 1 and 7.

• Phase 1: tyrosine concentration

  Tyrosine concentrations measured via blood spot analysis.

  Days 1 and 7.

• Phase 2: quantitative change in plasma amino acids at day 28

  Compare comprehensive plasma amino acid profiles at baseline and end of the trial.

  Phase 2, day 1 and day 28.

• Phase 2: gastrointestinal tolerance

  Questionnaires will be completed daily throughout Phase 2.

  Phase 2, days 1 - 28.

• Phase 2: change in 3-day diet record at end of study

  A 3-day diet record will be completed in the first 3 days and the last 3 days for comparison.

  Phase 2, days 1 - 3 and days 26 - 28.

• Phase 2: change in anthropometry at end of study

  Participants will be weighed and measured, a growth chart plotted (children only), and BMI calculated

  Phase 2, days 1 and 28.

• Phase 2: phenylalanine concentration

  Phenylalanine concentrations measured via blood spot analysis.

  Twice weekly for four weeks during phase 2

• Phase 2: tyrosine concentration

  Tyrosine concentrations measured via blood spot analysis.

  Twice weekly for four weeks during phase 2

• Phase 2: questionnaire data captured to evaluate change in taste perception

  Data captured to evaluate taste

  Phase 2, days 14 and 28.

• Phase 2: questionnaire data captured to evaluate change in smell perception

  Data captured to evaluate smell

  Phase 2, days 14 and 28.

• Phase 2: questionnaire data captured to evaluate change in texture perception

  Data captured to evaluate texture

  Phase 2, days 14 and 28.

Secondary Outcomes (2)

• Phase 1: questoinnaire evaluating subjective measures of satiety

  Phase 1, days 1 - 7.

• Phase 2: questoinnaire evaluating subjective measures of satiety

  Phase 2, days 1, 14 and 28.

Study Arms (1)

PKU Sphere

EXPERIMENTAL

Phase 1: 1 week To evaluate the acceptability of PKU Sphere during a short-term (1 week) period. Individuals with PKU will aim to consume a minimum of 30% of the medical food component of the diet as PKU Sphere. The amount will be assessed and advised on an individual basis. Phase 2: 4 weeks To evaluate longer-term acceptability and metabolic control in individuals with PKU consuming an agreed target of 50 - 100% of their medical food component of the diet as PKU Sphere for 4 weeks. Some individuals, particularly young children between the ages of 3 - 6 years, may require a 1 - 3 week build up period to reach target volume which will be assessed on an individual basis.

Dietary Supplement: PKU Sphere

Interventions

PKU Sphere DIETARY_SUPPLEMENT

PKU Sphere is a glycomacropeptide based alternative to amino-acid based products for the dietary management of phenylketonuria.

PKU Sphere

Eligibility Criteria

• Age: 3 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of PKU, identified by newborn screening (NBS) and treated since birth.
• Ages 3 years and above (this includes pregnant women who will be assessed on an individual basis by the Co-Principal Investigators).
• Currently or previously consuming a low phenylalanine diet supplemented with a medical food designed for the dietary management of PKU.
• English as primary language.
• Willingly given, written, informed consent from the participant, 18 or more years, or from the parent/caregiver for participants \< 18 years.
• Willingly given, written assent (if appropriate) for those \< 18 years.

You may not qualify if:

• Currently consuming 100% of the medical food component of the diet as PKU sphere.
• An inability, in the opinion of the investigator, to comply with the requirements of the protocol.
• Any other type of inherited metabolic disease.

Sponsors & Collaborators

• Vitaflo International, Ltd: lead
• Oregon Health and Science University: collaborator

Study Sites (1)

Oregon Health and Science University

Portland, Oregon, 97239, United States

Location

MeSH Terms

Conditions

Phenylketonurias

Condition Hierarchy (Ancestors)

Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Amino Acid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Sandra Van Calcar, PhD

  Oregon Health and Science University

  PRINCIPAL INVESTIGATOR

• Joyanna Hansen, PhD

  Oregon Health and Science University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Two phase study consisting of a 1 week taste introduction period followed by a 4 week period assessing acceptability and metabolic control.

Study Record Dates

• First Submitted: January 3, 2018
• First Posted: February 5, 2018
• Study Start: May 1, 2018
• Primary Completion: May 21, 2019
• Study Completion: May 21, 2019
• Last Updated: February 16, 2024

Record last verified: 2024-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)